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# Novartis’ Fabhalta for PNH Approved
- URL: https://www.fdaweb.com/novartis-fabhalta-for-pnh-approved/
- Published: 2023-12-06T12:00:00.000Z
- Updated: 2026-09-14T14:17:56.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155929

FDA has approved Novartis’ Fabhalta (iptacopan) as the first oral monotherapy to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). A company [statement](https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh?ref=fdaweb.com) says Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell destruction within and outside the blood vessels.

Novartis says the approval is based on the Phase 3 APPLY-PNH trial in patients with residual anemia despite prior anti-C5 treatment who switched to Fabhalta, which demonstrated superiority in hemoglobin improvement in the absence of red blood cell transfusions and in transfusion avoidance rate over patients who stayed on anti-C5 treatments. It says the approval also was supported by the Phase 3 APPOINT-PNH study in complement inhibitor-naïve patients.

Fabhalta may cause serious infections caused by encapsulated bacteria and is available only through a Risk Evaluation and Mitigation Strategy that requires vaccination for encapsulated bacteria.

The statement says Fabhalta was discovered at Novartis and is indicated to treat adults with PNH. It is in development for a range of complement-mediated diseases including immunoglobulin A nephropathy, C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome, the company says. It has received FDA breakthrough therapy and orphan drug designations.